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What is CRISPR?

Intellia is currently investigating CRISPR for gene editing, a new way to make changes to DNA to potentially treat genetic disease.

Your genome is your entire collection of genes that contain the DNA code for making proteins. Gene editing refers to a set of technologies, including CRISPR, which can be used to make DNA changes inside a cell. These cells can be edited inside the body (in vivo) or outside the body (ex vivo) from a patient or donor.

Adapted from a naturally occurring bacterial immune system, CRISPR, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, allows scientists to harness the power of natural DNA repair mechanisms

In the future, Intellia hopes to use its CRISPR technology to treat a variety of human diseases.

Types of Edits

Genetically driven diseases, caused by mutations (a change in the DNA sequence), come in many different forms. Intellia is applying and optimizing its CRISPR genome editing platform to address each of these types of mutations.

In Vivo

CRISPR is the therapy

Our systemic lipid nanoparticle (LNP)-based delivery system has unlocked treatment of genetic diseases to both selectively knock out disease-causing genes and restore necessary genetic functions by targeted insertion.

Ex Vivo

CRISPR creates the therapy

We are focused on engineering T cell therapies to provide them with particular enhanced attributes that may enable them to more effectively treat oncological and immunological diseases. Our approach is designed to improve safety and efficacy by engineering cell therapies that are more precise, potent and persistent.


How CRISPR/Cas9 Works

Genetics and CRISPR: 101

How Intellia's In Vivo Approach Works

How Intellia's Ex Vivo Approach Works

Intellia's Investigational ATTR Program

Intellia's Investigational HAE Program